A Safety, Tolerability, and Efficacy Study of VX-880 and VX-017 in Participants With Type 1 Diabetes

August 27, 2026 updated by: Vertex Pharmaceuticals Incorporated

A Master Protocol to Evaluate the Safety, Tolerability, and Efficacy of Islet Cell Replacement Therapies in Subjects Who Have Type 1 Diabetes Mellitus With Impaired Hypoglycemic Awareness and Severe Hypoglycemia: A Phase 1/2/3 Study of VX-880 and a Phase 1/2 Study of VX-017

This study will evaluate the safety, tolerability and efficacy of VX-880 infusion and VX-017 infusion in participants with type 1 diabetes (T1D) and impaired awareness of hypoglycemia (IAH) and severe hypoglycemia.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Estimated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
57

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact

The name and contact information for the person who can answer enrollment questions for a clinical study. Each location where the study is being conducted may also have a specific contact, who may be better able to answer those questions.

Study Locations

  • Canada
      • Edmonton, Canada
        • Recruiting
        • University of Alberta, Edmonton
      • Montreal, Canada
        • Recruiting
        • McGill University Health Centre
      • Toronto, Canada
        • Recruiting
        • Toronto General Hospital (TGH)
      • Vancouver, Canada
        • Recruiting
        • Vancouver General Hospital
  • France
      • Lille, France
        • Recruiting
        • CHU Lille
      • Strasbourg, France
        • Recruiting
        • Centre de recherche en Biomédecine de Strasbourg
  • Germany
      • Dresden, Germany
        • Recruiting
        • Dresden Center for Islet Transplantation
  • Italy
      • Milan, Italy
        • Recruiting
        • IRCCS Ospedale San Raffaele
  • Netherlands
      • Leiden, Netherlands
        • Recruiting
        • Leiden University
  • Norway
      • Oslo, Norway
        • Recruiting
        • Oslo University Hospital
  • Saudi Arabia
      • Riyadh, Saudi Arabia
        • Recruiting
        • King Abdullah International Medical Research Center (KAIMRC) - Riyadh - Endocrinology
      • Riyadh, Saudi Arabia
        • Recruiting
        • King Faisal Specialist Hospital & Research Centre - Riyadh - Endocrinology
  • Switzerland
      • Geneva, Switzerland
        • Completed
        • Hopiteaux Universitaires de Geneve
  • United Kingdom
      • Edinburgh, United Kingdom
        • Recruiting
        • Royal Infirmary of Edinburgh
      • London, United Kingdom
        • Recruiting
        • Cardiovascular, Metabolic Medicine and Sciences, King's College London
      • Newcastle upon Tyne, United Kingdom
        • Recruiting
        • The Newcastle upon Tyne Hospitals NHS Foundation Trust
    • Oxford
      • Headington, Oxford, United Kingdom
        • Recruiting
        • Churchill Hospital
  • United States
    • California
      • Duarte, California, United States, 91010
        • Recruiting
        • City Of Hope
      • San Francisco, California, United States, 94143
        • Recruiting
        • University of California San Francisco
    • Florida
      • Miami, Florida, United States, 33136
        • Recruiting
        • UHealth Diabetes Research Institute
    • Illinois
      • Chicago, Illinois, United States, 60637
        • Recruiting
        • University Of Chicago
      • Chicago, Illinois, United States, 60611
        • Completed
        • Northwestern Organ Transplant Center
    • Maryland
      • Baltimore, Maryland, United States, 21287
        • Completed
        • Johns Hopkins University
    • Massachusetts
      • Boston, Massachusetts, United States, 02114
        • Recruiting
        • Massachusetts General Hospital
    • Pennsylvania
      • Philadelphia, Pennsylvania, United States, 19104
        • Recruiting
        • Hospital of the University of Pennsylvania
      • Pittsburgh, Pennsylvania, United States, 15213
        • Recruiting
        • University of Pittsburgh Medical Center Montefiore
    • Texas
      • Dallas, Texas, United States, 75246
        • Recruiting
        • Baylor Scott and White Research Institute
    • Virginia
      • Richmond, Virginia, United States, 23298
        • Recruiting
        • VCU Medical Center, Richmond
    • Wisconsin
      • Madison, Wisconsin, United States, 53792
        • Recruiting
        • University Of Wisconsin

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years to 65 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Key Inclusion Criteria:

  • Clinical history of T1D with >=5 years of duration of insulin dependence
  • At least two episodes of documented severe hypoglycemia in the 12 months prior to enrollment
  • Stable diabetic treatment
  • Consistent use of continuous glucose monitor (CGM) for at least 3 months before Screening and willingness to use CGM for the duration of the study

Key Exclusion Criteria:

-Prior islet cell transplant, organ transplant, or cell therapy

Other protocol-defined Inclusion/Exclusion criteria may apply

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Non-Randomized
  • Interventional Model: Parallel Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: VX-880
Biological: VX-880
Infused into the hepatic portal vein.
Other Names:
  • Formerly known as STx-02
  • Zimislecel
Experimental: VX-017
Biological: VX-017
Infused into the hepatic portal vein.

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Time Frame
Part A: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
Time Frame: From VX-880 infusion to end of study (at least 5 years)
From VX-880 infusion to end of study (at least 5 years)
Parts B and C: Proportion of Participants who are Insulin Independent with Absence of Severe Hypoglycemic Events (SHEs)
Time Frame: 1 year after achieving insulin independence (following VX-880 infusion)
1 year after achieving insulin independence (following VX-880 infusion)
Part D: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
Time Frame: From VX-017 infusion to end of study (at least 5 years)
From VX-017 infusion to end of study (at least 5 years)

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Time Frame
Parts B and C: Proportion of Participants who are Insulin Independent
Time Frame: At 1 year after VX-880 infusion
At 1 year after VX-880 infusion
Parts B and C: Proportion of Participants Free from SHEs with a Glycated Hemoglobin (HbA1c) less than (<) 7.0%
Time Frame: SHEs: from Day 90 through 1 year (inclusive) after VX-880 infusion; HbA1c < 7% at 1 year after VX-880 infusion
SHEs: from Day 90 through 1 year (inclusive) after VX-880 infusion; HbA1c < 7% at 1 year after VX-880 infusion
Parts B and C: Change From Baseline in Glycated Hemoglobin (HbA1c)
Time Frame: At 1 year after VX-880 infusion
At 1 year after VX-880 infusion
Parts B and C: Proportion of Participants Who Maintain Insulin Independence for at least 1 year
Time Frame: From VX-880 infusion to end of study (at least 5 years)
From VX-880 infusion to end of study (at least 5 years)
Parts B and C: Safety and Tolerability as Assessed by Number of Participants With Adverse Events (AEs)
Time Frame: From VX-880 infusion up to end of study (at least 5 years)
From VX-880 infusion up to end of study (at least 5 years)
Part D: Change from baseline in peak C-peptide during mixed-meal tolerance test (MMTT)
Time Frame: At Day 90 after VX-017 infusion
At Day 90 after VX-017 infusion

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Vertex Pharmaceuticals Incorporated

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 29, 2021

Primary Completion (Estimated)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
December 31, 2027

Study Completion (Estimated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 31, 2031

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
March 3, 2021

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
March 3, 2021

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
March 8, 2021

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
September 1, 2026

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
August 27, 2026

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
August 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • VX20-880-101
  • 2024-513929-23-00 (Other Identifier: EU Trial (CTIS) Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

IPD Plan Description

Details on Vertex data sharing criteria and process for requesting access can be found at: https://www.vrtx.com/our-science/clinical-trials-data-sharing/

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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